Gene therapy involves introducing functional genes into patients' cells to treat diseases caused by defective genes. It works by replacing mutated genes with healthy copies, inactivating mutated genes, or introducing new genes to fight diseases. The process involves identifying the defective gene, cloning the normal gene, selecting target cells, and inserting the functional gene into the host DNA using viruses to deliver the new gene. Engineered cells are then injected into patients so the functional gene can correct the disorder.
Gene Therapy
2) What is Gene Therapy
An approach of treating diseases by either modifying the expressions of an individual’s genes or correction of abnormal genes.
3) This can be accomplished by:
Replacing a mutated gene that causes disease with a healthy copy of the gene.
Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
Introducing a new gene into the body to help fight a disease
4) NON VIRAL VECTOR SYSTEM
5) 1. PURE DNA CONSTRUCT
Direct introduction of pure DNA construct into target tissue.
Efficiency of DNA uptake by cells and expression rather low.
Consequently, large quantities of DNA have to be injected periodically.
6) 2. DNA MOLECULAR CONJUGATES
Commonly used synthetic conjugate is poly- L- lysine bound to specific target cell receptor.
Therapeutic DNA is then made to combine with the conjugate to form a complex.
It avoids lysosomal breakdown of DNA
7) 3. LIPOPLEXES
Lipid DNA complexes
DNA construct surrounded by artificial lipid layer.
Most of it gets degraded by lysosomes
8) 4. HUMAN ARTIFICIAL CHROMOSOME
Can carry a large DNA i.e., with one or more therapeutic genes
9) References :-
Biotechnology by U. Satyanarayana and U. Chakrapani, First edition, books and allied (p) Ltd,2005. Page no.213-226.
NPTEL-Bio Technology – Genetic Engineering and applications “MODULE 5-LECTURE 1 GENE TRANSFER TECHNIQUES : BIOLOGICAL METHODS .” Page no : 3-22.
"Gene Therapy" is a technique used to treat a disease or disorder by inducing the therapeutic gene into the vector, and the vector into the affected organism. So that, the therapeutic proteins expressed will vanish the affected proteins.
GENE THERAPY: TYPES, METHODS, FACTORS AND STANDARDS AND ITS APPLICATION IN HEALTHCARE FIELD
INVIVO THERAPY AND EXVIVO THERAPY
CHEMICAL AND PHYSICAL METHODS TO CARRY ON GENE THERAPY
DEFECTIVE GENE IDENTIFICATION IN GENE THERAPY AND TREATMENT OF GENETICALLY AFFECTED GENE BY GENE THERAPY
Gene Therapy
2) What is Gene Therapy
An approach of treating diseases by either modifying the expressions of an individual’s genes or correction of abnormal genes.
3) This can be accomplished by:
Replacing a mutated gene that causes disease with a healthy copy of the gene.
Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
Introducing a new gene into the body to help fight a disease
4) NON VIRAL VECTOR SYSTEM
5) 1. PURE DNA CONSTRUCT
Direct introduction of pure DNA construct into target tissue.
Efficiency of DNA uptake by cells and expression rather low.
Consequently, large quantities of DNA have to be injected periodically.
6) 2. DNA MOLECULAR CONJUGATES
Commonly used synthetic conjugate is poly- L- lysine bound to specific target cell receptor.
Therapeutic DNA is then made to combine with the conjugate to form a complex.
It avoids lysosomal breakdown of DNA
7) 3. LIPOPLEXES
Lipid DNA complexes
DNA construct surrounded by artificial lipid layer.
Most of it gets degraded by lysosomes
8) 4. HUMAN ARTIFICIAL CHROMOSOME
Can carry a large DNA i.e., with one or more therapeutic genes
9) References :-
Biotechnology by U. Satyanarayana and U. Chakrapani, First edition, books and allied (p) Ltd,2005. Page no.213-226.
NPTEL-Bio Technology – Genetic Engineering and applications “MODULE 5-LECTURE 1 GENE TRANSFER TECHNIQUES : BIOLOGICAL METHODS .” Page no : 3-22.
"Gene Therapy" is a technique used to treat a disease or disorder by inducing the therapeutic gene into the vector, and the vector into the affected organism. So that, the therapeutic proteins expressed will vanish the affected proteins.
GENE THERAPY: TYPES, METHODS, FACTORS AND STANDARDS AND ITS APPLICATION IN HEALTHCARE FIELD
INVIVO THERAPY AND EXVIVO THERAPY
CHEMICAL AND PHYSICAL METHODS TO CARRY ON GENE THERAPY
DEFECTIVE GENE IDENTIFICATION IN GENE THERAPY AND TREATMENT OF GENETICALLY AFFECTED GENE BY GENE THERAPY
Gene therapy refers to the insertion of genetic material to correct a genetic defect.
In gene therapy, a "normal" gene is inserted into the genome to replace an "abnormal," disease-causing gene
A good comprehensive review of gene delivery and gene therapy. especially for master of pharmacy 2nd-semester students as per the PCI syllabus of subject Molecular pharmaceutics.
List of contents under this ppt :
{A} GENE THERAPY
(1) Definition
(2) Introduction
(3) History
(4) Ex-Vivo gene therapy
(5) In-Vivo gene therapy
(6) Germline gene therapy
(7) Advantages of gene therapy
(8) Disadvantages of gene therapy
(9) Potential target diseases for gene therapy
a. inherited disorders :- ADA SCID, Chronic granulomatous, Hemophelia
b. Cancer
{B} GENE DELIVERY
(1) Definition
(2) Introduction
(3) Types of vectors
a. Viral :- Retrovirus, Adenovirus, Adeno associated virus, Herps simplex virus
b. Non viral :-
Physical methods - Gene gun, Microinjection, Electroporation, Sonoporation
Chemical methods - Oligonucleotides, Lipoplexes, Polyplexes, Dendrimers, Nanoparticles.
This presentation is all about biotechnology. It is about the basic aspects of Biotechnology and covers a lot of topics under biotechnology, recombinant DNA technology. This is specifically for the HSC students of Mumbai. I hope that it helps.
The French Revolution, which began in 1789, was a period of radical social and political upheaval in France. It marked the decline of absolute monarchies, the rise of secular and democratic republics, and the eventual rise of Napoleon Bonaparte. This revolutionary period is crucial in understanding the transition from feudalism to modernity in Europe.
For more information, visit-www.vavaclasses.com
The Roman Empire A Historical Colossus.pdfkaushalkr1407
The Roman Empire, a vast and enduring power, stands as one of history's most remarkable civilizations, leaving an indelible imprint on the world. It emerged from the Roman Republic, transitioning into an imperial powerhouse under the leadership of Augustus Caesar in 27 BCE. This transformation marked the beginning of an era defined by unprecedented territorial expansion, architectural marvels, and profound cultural influence.
The empire's roots lie in the city of Rome, founded, according to legend, by Romulus in 753 BCE. Over centuries, Rome evolved from a small settlement to a formidable republic, characterized by a complex political system with elected officials and checks on power. However, internal strife, class conflicts, and military ambitions paved the way for the end of the Republic. Julius Caesar’s dictatorship and subsequent assassination in 44 BCE created a power vacuum, leading to a civil war. Octavian, later Augustus, emerged victorious, heralding the Roman Empire’s birth.
Under Augustus, the empire experienced the Pax Romana, a 200-year period of relative peace and stability. Augustus reformed the military, established efficient administrative systems, and initiated grand construction projects. The empire's borders expanded, encompassing territories from Britain to Egypt and from Spain to the Euphrates. Roman legions, renowned for their discipline and engineering prowess, secured and maintained these vast territories, building roads, fortifications, and cities that facilitated control and integration.
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Roman architecture and engineering achievements were monumental. They perfected the arch, vault, and dome, constructing enduring structures like the Colosseum, Pantheon, and aqueducts. These engineering marvels not only showcased Roman ingenuity but also served practical purposes, from public entertainment to water supply.
Operation “Blue Star” is the only event in the history of Independent India where the state went into war with its own people. Even after about 40 years it is not clear if it was culmination of states anger over people of the region, a political game of power or start of dictatorial chapter in the democratic setup.
The people of Punjab felt alienated from main stream due to denial of their just demands during a long democratic struggle since independence. As it happen all over the word, it led to militant struggle with great loss of lives of military, police and civilian personnel. Killing of Indira Gandhi and massacre of innocent Sikhs in Delhi and other India cities was also associated with this movement.
2024.06.01 Introducing a competency framework for languag learning materials ...Sandy Millin
http://sandymillin.wordpress.com/iateflwebinar2024
Published classroom materials form the basis of syllabuses, drive teacher professional development, and have a potentially huge influence on learners, teachers and education systems. All teachers also create their own materials, whether a few sentences on a blackboard, a highly-structured fully-realised online course, or anything in between. Despite this, the knowledge and skills needed to create effective language learning materials are rarely part of teacher training, and are mostly learnt by trial and error.
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June 3, 2024 Anti-Semitism Letter Sent to MIT President Kornbluth and MIT Cor...Levi Shapiro
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The US House of Representatives is deeply concerned by ongoing and pervasive acts of antisemitic
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• The Committee on Education and the Workforce has been investigating your institution since December 7, 2023. The Committee has broad jurisdiction over postsecondary education, including its compliance with Title VI of the Civil Rights Act, campus safety concerns over disruptions to the learning environment, and the awarding of federal student aid under the Higher Education Act.
• The Committee on Oversight and Accountability is investigating the sources of funding and other support flowing to groups espousing pro-Hamas propaganda and engaged in antisemitic harassment and intimidation of students. The Committee on Oversight and Accountability is the principal oversight committee of the US House of Representatives and has broad authority to investigate “any matter” at “any time” under House Rule X.
• The Committee on Ways and Means has been investigating several universities since November 15, 2023, when the Committee held a hearing entitled From Ivory Towers to Dark Corners: Investigating the Nexus Between Antisemitism, Tax-Exempt Universities, and Terror Financing. The Committee followed the hearing with letters to those institutions on January 10, 202
Model Attribute Check Company Auto PropertyCeline George
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2. • Definition of gene therapy:
“ Novel approach to treat,cure or ultimately
prevent a disease by changing the
expression of a person’s genes”
Dr. Padmesh,V,Slideshare.net
4. Gene Therapy
• Tell me how do you want to change
– Replacing a mutated gene that causes
disease with a healthy copy of the gene.
– Inactivating, or “knocking out,” a mutated
gene that is functioning improperly.
– Introducing a new gene into the body to help
fight a disease.
Dr. Padmesh V, Slideshare.net
5. STEPS IN GENE THERAPY:
2. Identification of the defective gene.
3. Cloning of normal healthy gene.
4. Identification of target cell / tissue / organ.
5. Insertion of the normal functional gene into the host
DNA.
METHOD:
Introduction of FUNCTIONAL GENES into appropriate cells
Transferred gene (TRANSGENE) encodes & produces proteins
The Proteins encoded by Transgene corrects the disorder
Dr. Padmesh V, Slideshare.net
6. Cloned gene
(normal allele) 1 An RNA version of
a normal human
gene is inserted
into a retrovirus.
RNA genome of virus
Retrovirus
2 Bone marrow cells
are infected with
the virus.
3 Viral DNA carrying the
human gene inserts into
the cell’s chromosome.
Bone marrow
cell from the patient
4 The engineered
cells are injected
into the patient.
Bone
marrow
7. Cloned gene
(normal allele) 1 An RNA version of
a normal human
gene is inserted
into a retrovirus.
RNA genome of virus
Retrovirus
8. 2 Bone marrow cells
are infected with
the virus.
3 Viral DNA carrying the
human gene inserts into
the cell’s chromosome.
Bone marrow
cell from the patient
4 The engineered
cells are injected
into the patient. Bone
marrow
9. ore
e no m !
u ar nant!!
Yo m i
Do
Google images
Editor's Notes
Figure 12.10 One type of gene therapy procedure
Figure 12.10_1 One type of gene therapy procedure (part 1)
Figure 12.10_2 One type of gene therapy procedure (part 2)
